Iowa City, IANCT06663358Now EnrollingIRB Ready

Fabry Disease Clinical Trial in Iowa City, IA

Access cutting-edge fabry disease treatment through this clinical trial at a research site in Iowa City. Study-provided care at no cost to qualified participants.

Sponsored by Chiesi Farmaceutici S.p.A.

Quick Self-Assessment

See if you qualify for this Iowa City location

Preparing your pre-screening questions…

Expert Care in Iowa City

Access fabry disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related fabry disease treatment provided free

Apply for This Iowa City Location

Check if you qualify for this fabry disease clinical trial in Iowa City, IA

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Iowa City

    Convenient for IA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Iowa City site if eligible
  4. 4Begin participation

About This Fabry Disease Study in Iowa City

A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

Sponsor: Chiesi Farmaceutici S.p.A.

Who Can Participate

Inclusion Criteria

Male or female aged \> 18 years of age at the time of consent.
Genetically confirmed diagnosis of Fabry disease.
Either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease.
No contraindications for cardiac magnetic resonance imaging (cMRI)
Informed consent form (ICF) signed and dated indicating the individual has been informed of and agreed to all pertinent aspects of the study and is willing to comply with all study requirements, including completion of electronic patient reported outcomes (ePROs).
Cardiac Cohort:
Evidence of Fabry disease (FD)-related heart disease including one or more of the following:
Left ventricular hypertrophy (LVH) measured by left ventricular mass index (LVMI) (g/m2) elevation above age/sex specific reference ranges.
Posterior septum wall thickness (e.g., \>=13mm) not explained by other factors (e.g., hypertension)
Low native T1 mapping on cMRI.
Typical Fabry-like scar on cMRI
Participants can receive cardiac magnetic resonance imaging (cMRI) with gadolinium enhancement as part of their SoC.
Estimated glomerular filtration rate (eGFR) \>45 mL/min/1.73 m2, assessed within the prior 6 months.
Naïve Cohort:
Most recent eGFR\>45 mL/min/1.73 m2, assessed within prior 6 months.
Male participants should have abnormal elevation in plasma lysoGb3 as assessed within 6 months prior to enrolment.
Long-Term Cohort:
Participants previously enrolled in the open label study CLI-06657AA1-04 (Previously PB-102-F60) (using pegunigalsidase alfa at a dose of 1mg/kg every 2 weeks) who have initiated or plan to initiate commercial pegunigalsidase alfa (Elfabrio®).

Exclusion Criteria

Contraindication to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium contrast agent that is not managed by the use of premedication.
Pregnant at the time of enrolment.
Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
Active participation in any interventional study for Fabry disease
Treatment regimen at the time of enrolment in the study is different from the approved 1mg/kg every two weeks (note if regimen subsequently changes during the prospective part of the study, the participants can remain in the study)
Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg/kg administered every 4 weeks.
Cardiac Cohort:
History of acute myocardial infarction or congestive heart failure with reduced left ventricular (LV) ejection fraction of less than 35%.
Cerebral vascular accident (CVA) in the prior 6 months.
Chronic liver cirrhosis.
FD-unrelated heart disease (e.g., scarring due to myocardial infarction, symptomatic occlusive coronary artery disease, moderate valvular heart disease not thought to be Fabry related).
The participant is or has been treated with any investigational drug for Fabry disease within 6 months of study start or investigational gene therapy for Fabry disease at any time point in the past.
Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI at any prior cMRI.
Naïve Cohort:
Prior exposure to a FD therapy (Replagal®, Fabrazyme®, and Galafold®) at any time point.
Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI on any prior cMRI

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Iowa City?

Yes, this clinical trial (NCT06663358) has an active research site in Iowa City, IA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Fabry Disease Treatment Options in Iowa City, IA

If you're searching for fabry disease treatment options in Iowa City, IA, this clinical trial (NCT06663358) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Iowa City research site is actively enrolling participants for this clinical trial. You'll receive care from experienced fabry disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all fabry disease clinical trials near you to find additional studies recruiting in your area.

More Ankylosing Spondylitis Trials in Iowa City, IA

See all ankylosing spondylitis clinical trials recruiting in Iowa City — not just this study.

Browse Ankylosing Spondylitis Trials in Iowa City →

Browse More Trials by Condition

Ankylosing Spondylitis TrialsFabry Disease TrialsFanconi Anemia TrialsAll Conditions →

Ready to Join in Iowa City?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Iowa City, IA